BioMarin Pharmaceutical’s acquisition of Amicus Therapeutics is a marriage of two rare disease biotech companies. Amicus’s two commercialized products, for the enzyme deficiencies Fabry disease and Pompe disease, are each projected to become blockbuster sellers.
Sanofi, which markets the two primary treatments for Pompe disease, was trying to add a third one by licensing rights to a Maze Therapeutics drug candidate with a different therapeutic approach. A Federal Trade Commission complaint alleges the deal amounts to a monopoly player taking out its competition.
Zelis highlights how employers, payers, and consumers are integrating digital tools to manage healthcare costs.
Sanofi dominates the market for therapies that treat Pompe disease, a rare enzyme deficiency. The FDA has approved an Amicus Therapeutics combination treatment for patients inadequately served by the Sanofi products.
Sanofi is acquiring rights to a Maze Therapeutics drug candidate for Pompe disease that could give the pharmaceutical giant another option to offer patients who have the rare metabolic disorder. In February, the drug posted positive data from a Phase 1 study.
Sanofi won European Commission approval for two therapies that treat rare enzyme deficiencies. Xenpoyzme is the first treatment for acid sphingomyelinase deficiency (ASMD) while Nexviadyme is a successor to a blockbuster Sanofi drug for Pompe disease.
Amicus Therapeutics CEO John Crowley said the totality of data support the Pompe disease therapy’s submission to the FDA. That rolling application is on track for completion in the second quarter.
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.